Objective
Chronic kidney disease (CKD) is a leading cause of global mortality, affecting ~13% of global population. Inherited kidney diseases (IKD) are estimated to account for a 30% of CKD cases. Despite the unprecedented development of genetic medicines in the past decades, to date no kidney gene therapy intervention is available. Renal gene therapies have encountered two main barriers: (1) its complex anatomy and function has hampered efficient and specific delivery of genetic therapies to the kidney, and (2) many IKDs are caused by mutations in large genes, and therefore, traditional gene supplementation strategies using efficient and safe in vivo vectors (such as adeno-associated viral vectors or AAV) cannot be developed due to the limited packaging capacity. The main goal of this proposal is to create solutions for these challenges by developing novel gene therapy vectors with improved kidney targeting and genome editing strategies to correct the genetic defect in large genes that cause IKDs.
Recent advances in the field of discovery and engineering of gene delivery vehicles combined with CRISPR-based gene editing technologies have opened the avenue towards developing efficient and safe gene editing therapies. In this proposal, we will develop in vivo genome editing approaches for Autosomal Dominant Polycystic Kidney Disease (ADPKD), the most frequent IKD with an incidence of 1:1,000. For this, we will develop novel AAV gene delivery vectors, a technology that has been clinically validated to target several tissues, enabling an efficient delivery of CRISPR editors to the kidney. Additionally, CRISPR approaches will be designed to restore the physiological expression of the mutated PKD1 gene that causes ADPKD and tested in APDKD mouse and pig models in vivo and in patient-derived kidney organoid models in vitro. The completion of this proposal will enable the generation of personalized and universally applicable in vivo gene editing therapies for ADPKD patients.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- medical and health sciences basic medicine anatomy and morphology
- social sciences sociology demography mortality
- medical and health sciences medical biotechnology genetic engineering gene therapy
- natural sciences biological sciences genetics mutation
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.1.1 - European Research Council (ERC)
MAIN PROGRAMME
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-ERC - HORIZON ERC Grants
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2025-STG
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
31008 Pamplona
Spain
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.